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外泌体在神经退行性疾病中的应用:诊断与靶向治疗。

Exosomes for neurodegenerative diseases: diagnosis and targeted therapy.

机构信息

State Key Laboratory of Oral Diseases, National Clinical Research Center for Oral Diseases, West China Hospital of Stomatology, Sichuan University, Chengdu, 610041, China.

出版信息

J Neurol. 2024 Jun;271(6):3050-3062. doi: 10.1007/s00415-024-12329-w. Epub 2024 Apr 12.


DOI:10.1007/s00415-024-12329-w
PMID:38605227
Abstract

PURPOSE OF REVIEW: Neurodegenerative diseases are still challenging clinical issues, with no curative interventions available and early, accurate diagnosis remaining difficult. Finding solutions to them is of great importance. In this review, we discuss possible exosomal diagnostic biomarkers and explore current explorations in exosome-targeted therapy for some common neurodegenerative diseases, offering insights into the clinical transformation of exosomes in this field. RECENT FINDINGS: The burgeoning research on exosomes has shed light on their potential applications in disease diagnosis and treatment. As a type of extracellular vesicles, exosomes are capable of crossing the blood - brain barrier and exist in various body fluids, whose components can reflect pathophysiological changes in the brain. In addition, they can deliver specific drugs to brain tissue, and even possess certain therapeutic effects themselves. And the recent advancements in engineering modification technology have further enabled exosomes to selectively target specific sites, facilitating the possibility of targeted therapy for neurodegenerative diseases. The unique properties of exosomes give them great potential in the diagnosis and treatment of neurodegenerative diseases, and provide novel ideas for dealing with such diseases.

摘要

目的综述:神经退行性疾病仍然是具有挑战性的临床问题,目前尚无有效的治疗干预措施,早期准确诊断仍然困难。找到解决这些问题的方法非常重要。在这篇综述中,我们讨论了一些常见神经退行性疾病中潜在的外泌体诊断生物标志物,并探讨了目前针对外泌体的治疗探索,为该领域中外泌体的临床转化提供了一些见解。

最近的发现:对外泌体的研究蓬勃发展,为其在疾病诊断和治疗中的应用提供了新的思路。作为一种细胞外囊泡,外泌体能够穿透血脑屏障,并存在于各种体液中,其成分可以反映大脑的病理生理变化。此外,它们可以将特定的药物递送到脑组织中,甚至具有一定的治疗作用。最近工程修饰技术的进步进一步使外泌体能够选择性地靶向特定部位,为神经退行性疾病的靶向治疗提供了可能。外泌体的独特性质使其在神经退行性疾病的诊断和治疗中具有巨大的潜力,为应对这些疾病提供了新的思路。

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Exosomes for neurodegenerative diseases: diagnosis and targeted therapy.

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引用本文的文献

[1]
Advances in Neurodegenerative Disease Therapy: Stem Cell Clinical Trials and Promise of Engineered Exosomes.

CNS Neurosci Ther. 2025-9

[2]
Exosomes: innovative biomarkers leading the charge in non-invasive cancer diagnostics.

Theranostics. 2025-4-13

[3]
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[4]
Skeletal myotubes expressing ALS mutant SOD1 induce pathogenic changes, impair mitochondrial axonal transport, and trigger motoneuron death.

Mol Med. 2024-10-25

本文引用的文献

[1]
miR-100a-5p-enriched exosomes derived from mesenchymal stem cells enhance the anti-oxidant effect in a Parkinson's disease model via regulation of Nox4/ROS/Nrf2 signaling.

J Transl Med. 2023-10-24

[2]
Evaluation of ferritin and TfR level in plasma neural-derived exosomes as potential markers of Parkinson's disease.

Front Aging Neurosci. 2023-9-19

[3]
FTO-targeted siRNA delivery by MSC-derived exosomes synergistically alleviates dopaminergic neuronal death in Parkinson's disease via m6A-dependent regulation of ATM mRNA.

J Transl Med. 2023-9-22

[4]
Impact of the Drug Loading Method on the Drug Distribution and Biological Efficacy of Exosomes.

AAPS PharmSciTech. 2023-8-8

[5]
Soluble mutant huntingtin drives early human pathogenesis in Huntington's disease.

Cell Mol Life Sci. 2023-8-3

[6]
MicroRNA-23a-3p Is Upregulated in Plasma Exosomes of Bulbar-onset ALS Patients and Targets ERBB4.

Neuroscience. 2023-8-1

[7]
Small RNA sequencing of circulating small extracellular vesicles microRNAs in patients with amyotrophic lateral sclerosis.

Sci Rep. 2023-4-4

[8]
Signature of miRNAs derived from the circulating exosomes of patients with amyotrophic lateral sclerosis.

Front Aging Neurosci. 2023-2-10

[9]
Circular RNAs in Parkinson's Disease: Reliable Biological Markers and Targets for Rehabilitation.

Mol Neurobiol. 2023-6

[10]
Employing nanoparticle tracking analysis of salivary neuronal exosomes for early detection of neurodegenerative diseases.

Transl Neurodegener. 2023-2-7

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